The patent badge is an abbreviated version of the USPTO patent document. The patent badge does contain a link to the full patent document.
The patent badge is an abbreviated version of the USPTO patent document. The patent badge covers the following: Patent number, Date patent was issued, Date patent was filed, Title of the patent, Applicant, Inventor, Assignee, Attorney firm, Primary examiner, Assistant examiner, CPCs, and Abstract. The patent badge does contain a link to the full patent document (in Adobe Acrobat format, aka pdf). To download or print any patent click here.
Patent No.:
Date of Patent:
Nov. 06, 2018
Filed:
Aug. 30, 2017
Applicant:
Samsung Life Public Welfare Foundation, Yongsan-gu, KR;
Inventors:
Byung-Ok Choi, Seoul, KR;
Young Bin Hong, Seoul, KR;
Assignee:
SAMSUNG LIFE PUBLIC WELFARE FOUNDATION, Seoul, KR;
Attorney:
Primary Examiner:
Int. Cl.
CPC ...
C12N 15/113 (2010.01); A61K 9/127 (2006.01); A61K 31/713 (2006.01); C12N 15/11 (2006.01);
U.S. Cl.
CPC ...
C12N 15/113 (2013.01); A61K 9/1272 (2013.01); A61K 31/713 (2013.01); C12N 15/11 (2013.01); C12N 15/1138 (2013.01); C12N 2310/14 (2013.01); C12N 2320/34 (2013.01);
Abstract
An siRNA for specifically targeting a PMP22 mutant gene and a pharmaceutical composition for preventing or treating Charcot Marie Tooth disease, which includes the same, are provided. According to the present invention, it is confirmed that selective suppression of a PMP22 mutant allele by a non-viral delivery system of siRNA may improve demyelinating neuropathic symptoms of CMT in vivo, enhance a motor ability and increase a volume of muscle. Therefore, the siRNA may be used in a useful method for treating various dominantly inherited peripheral neuropathies including CMT.